Exam 2 : Lecture Interventional Flashcards
Other terms for interventional
Clinical trial Clinical study Experimental study Human study Investigational study
Interventional studies are MORE rigorous in ability to show cause-and-effect . T or F
True
Interventional studies CANT demonstrate causation. T or F?
False. They CAN
Prior to human investigation this level used animal research “bench”
Pre-clinical
In this phase we:
- Assess drug target actions and pharmacokinetics in non therapeutic/non-diagnostic doses
- Healthy volunteers (diseased in oncology)
- Very small numbers
- Very short duration
Phase 0
In this phase we:
1. Assess safety/tolerance and pharmacokinetics of one or more dosages
2. Healthy or disease volunteers (depends on disease)
3. Small numbers (20-80)
Short duration (just a few weeks)
Phase 1
In this phase we:
- Asses effectiveness
- Disease volunteers
- Larger number (100-300)
- Short to medium duration (few weeks to a few months)
Phase 2
In this phase we:
- Asses effectiveness
- Diseased volunteers
- superiority
- noninferiority
- equivalency - Larger number (300-500)
- Longer duration (few months to a year +)
Phase 3
Phase three can be run multiple times, T or F?
True. It can be run multiple times
If the drug isn’t yet on the market, what phase is it in?
Phase 3
Every time phase three is tested, it must be successful to move on to stage 4. T or F?
False. It just needs to majoritively test positive
In this phase we:
- Assess long-term safety, effectiveness, optimal use
- Diseased voulnteers
- Population (100,000)
- Longer duration (many years to decades)
Phase 4
FDA approval phase
Phase 4
What are some advantages of interventional trials
- Cause precedes effect (can demonstrate causation)
2. Only design-family used by FDA for “approval” process
Disadvantages of interventional trials?
Cost
Complexity
Ethical considerations
Generalizability
This interventional study divides subjects exclusively int o2 or more groups. Asks a single question. What study design is it?
Simple
This interventional study design divides subjecting into 2 or more groups and then further sub divides each of the groups into 2 more additional groups. Tests multiple hypothesis. What design is this?
Factorial
What are some benifits and risks to using factorial interventional studies?
Improves efficiency for answering clinical questions Increases study population sample size Increases complexity Increases risk of drop outs May restrict generalizability of results
Are simple and factorial study designs also parallel?
Yes
NO SWITCHING
In this interventional study design groups serve as their own control by crossing over from one intervention to another during the study. What design is this?
Cross-over (self control)
Which interventional design has a wash out phase?
Cross-over
Disadvantages of cross-over design?
- Only suitable for long-term conditions which are not curable or which treatment provides short-term relief
- Duration of study for each subject is longer
- Carry-over effects during cross-over
- Treatment by period interaction
- Smaller number requirement only applicable if within subjects variation less than between subjects variation
- Complexity in data analysis
Patient oriented endpoint (POEM’s)
Death
Stroke
Hospitalization
Preventing need for dialysis
Disease oriented endpoints (DOE’s)
Blood pressure
Cholesterol
Change in SCr
Sample selection: Patients dont have an equal probability of being selected. Ex: all of the 8 am epi class is selected for group 1 and all of micro is group 2
Non-random
What table is customarily use to show group characteristics?
Table 1
Equality of groups is guaranteed in randomization. T or F?
False
Examples of forms of randomization
Simple
Blocked
Stratified
Simple randomization
Equal probability for allocation within one of the study groups
Blocked randomization
Every 20 you peak at the groups to make sure they are equal
Ensures balance within each intervention group
Stratified randomization
Ensures balance with known confounding variables
Ex: equal number of ages in both groups, check as you go.
Types of masking
Single
Double
Single mask
Study subjects not informed which intervention group they are in, yet investigators are permitted to know
Fine for: objective (blood pressure monitoring extc..)
Double blind
Neither investigator nor study subjects are informed which intervention group subjects are in
-post surveys are used to assess credibility (adequacy) of double blind
Open label
Study subjects and researchers know what intervention is being received
What can be used to assess adequacy of blinding
Post hoc survey’s
Plaebebo
Inert treatments made to look identical in all aspects to the active treatment
Double dummy
More than 1 placebo
Hawthorne-effect
Study subjects change their behavior solely due to awareness of being studied
Post hoc sub group analysis is always accepted as appropriate. T or F?
False. It’s only appropriate when its been planned for.
How doe we manage drop-outs/lost to follow ups?
Intention to treat
Intentions to treat
Use last known assessment
Convert all subsequent yet missed assessments for a subject to a null effect
Per-protocol or efficacy analysis?
Ignore drop outs
Include only the compliant or completing subjects
Assess adherence (compliance) how
Drug levels
Pill count
Bottle counter tops
Methods of improving adherence?
Frequent follow up visits
Treatment alarms
Medication blister packs or dosage containers
